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New gene editing strategies developed for Duchenne muscular dystrophy

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IMAGE: In a mouse model and in human heart muscle cells, researchers used gene editing to modify specific DNA sequences and restore dystrophin production in mutant dystrophin genes.
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Credit: UT Southwestern Medical Center
DALLAS - April 30, 2021 - UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal
Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases. ....

United States , Cristina Rodriguez Caycedo , Roberta Welch , Eric Olson , Alexa Mireault , Ayhan Atmanli , Francesco Chemello , Andreasc Chai , Efrain Sanchez Ortiz , Rhonda Bassel Duby , National Institutes Of Health , Roberta Welch Foundation , Research On Cardiac Birth Defects , Willie Nelson Professorship In Stem Cell Research , Research Institute Of Texas , Science Advances , Distinguished Chair , Pogue Distinguished Chair , Cardiac Birth Defects , Willie Nelson Professorship , Stem Cell , Ning Liu , National Institutes , Senator Paul , Muscular Dystrophy Specialized Research Center , Cancer Prevention ,

Digging deep for differences in Duchenne muscular dystrophy

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IMAGE: A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD).
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Credit: UT Southwestern Medical Center
DALLAS - Dec. 21, 2020 - A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchenne muscular dystrophy (DMD) in humans. The findings, published online recently in
PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development. ....

United States , Francesco Chemello , Roberta Welch , Eric Olson , Rhonda Bassel Duby , Johnr Mcanally , Zhaoning Wang , American Heart Association , Harrys Moss Heart , Hamon Center , National Institutes Of Health , Roberta Welch Foundation , Willie Nelson Professorship In Stem Cell Research , Wellstone Muscular Dystrophy Center , Research On Cardiac Birth Defects , Regenerative Science , Senator Paul , Muscular Dystrophy Cooperative Research Center , Pogue Distinguished Chair , Cardiac Birth Defects , Distinguished Chair , Willie Nelson Professorship , Stem Cell , National Institutes , Heart Trust , ஒன்றுபட்டது மாநிலங்களில் ,